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  • Writer's pictureDr Edin Hamzić

💊 Shortly About Midostaurin

Updated: Jan 26

What is Midostaurin? What are other names for Midostaurin?

Midostaurin is the first approved oral tyrosine kinase inhibitor (TKI), sold under the commercial name Rydapt & Tauritmo by Novartis Pharmaceuticals; it was approved on April 28, 2017 [citation].

Midostaurin is combined with standard intensive chemotherapy for adult patients with newly diagnosed FLT3-mutated AML [citation]. In addition, Midostaurin is also used to treat aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, or mast cell leukemia [citation].

Commercial names by which the midostaurin is distributed are Ryadapt and Tauritmo.

How Midostaurin works? What is the mode of action for Midostaurin?

Midostaurin is prescribed to newly diagnosed patients with an FLT3-activating mutation, either FLT3-ITD or FLT3-TKD. It is a tyrosine kinase inhibitor that works in a way to block the action of the abnormal protein encoded as a consequence of FLT3-ITD or FLT3-TKD mutations to prevent tumor growth.

Midostaurin and its primary active metabolites CGP62221 and CGP52421 inhibit the activity of KIT (wild type and D816V mutant), FLT3, PDGFR, CSFͲ1R, ROS1, members of the protein kinase C family, and several other receptor tyrosine kinases including FGFR and VEGFR2 [citation].

Inhibition of FLT3 receptor signaling cascades leads to apoptosis of target leukemia cells expressing target receptors and mast cells, in addition to its antiproliferative activity toward multiple cancer cell lines [citation].

How is Midostaurin applied?

Midostaurin is taken orally twice daily [citation].

When is Midostaurin used?

Midostaurin is prescribed to newly diagnosed adult patients with FLT3-mutated AML [citation].

Who produces Midostaurin?

As mentioned above, Midostaurin is produced by Novartis Pharmaceuticals, Inc., Basel, Switzerland.


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